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The Rise of RNA: A New Target for Therapeutic Agents

The Rise of RNA: A New Target for Therapeutic Agents

University at Albany-SUNY

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Poor RNA. It has never managed to attract the scientific spotlight that DNA has, and the molecule seems destined to be forever considered a mere supporting player in the great drama of the molecular biology of life.

Not if The RNA Institute at the University at Albany, part of the State University of New York (SUNY), has anything to say about it, however. The Institute was founded in 2010 on the conviction that RNA is not a mere errand boy for DNA, but is a promising target for therapies that might treat diseases ranging from cancer to depression.

Genetic dogma holds that DNA, found in cell nuclei, contains the genetic code—the book of life and the blueprint for a human being. In less soaring rhetoric, however, DNA is simply a twisting staircase of a molecule (a double helix) that holds instructions, called genes, for the production of proteins, including structural ones like the actin and myosin that make muscles move as well as enzymes like pepsin that digest food and others that regulate cell development.

A century of pharmaceutical development has focused on proteins, such as by providing the protein insulin to diabetics. More recently, genetic medicine has targeted DNA to treat hereditary diseases, such as by replacing the faulty gene that causes thalassemia or by blocking the effects of mutated genes that underlie cancers. Nothing therapeutic targeted RNA. Its job seemed to be merely to carry DNA's instructions to cellular structures called ribosomes that produce the proteins. Why shoot the messenger?

The RNA Institute, directed by Paul F. Agris, professor of biological sciences and chemistry, is premised on a paradigm that sees RNA as much more important than a mere messenger. Scientists have recently shown that cells make countless RNAs that control protein production, a feat they manage by acting as the on/off switch for genes. Since disease results when proteins are not made correctly or in the quantity required by the cell, it might be possible to target RNA, inducing it to shut off disease-causing genes, for instance, or rev up laggard ones in order to provide proteins in the quantities a cell needs.

RNA, since it is involved in multiple steps leading to protein production, therefore has the potential to be the target of a novel class of therapeutic agents. Scientists at The RNA Institute are working to develop RNA-based medications, vaccines, and diagnostics for breast cancer; for drug-resistance in bacterial and viral infections, including MRSA (Staphylococcus aureus), drug resistant tuberculosis, and HIV; neurodegenerative and neuromuscular disorders such as amyotrophic lateral sclerosis (Lou Gehrig's disease) and neurofibromatosis ("elephant man disease"); depression; orphan diseases such as mitochondrial diseases and Sjögren's syndrome; and neuropathies.

RNA-based drug discovery begins with RNA target identification, characterization, and validation; moves on to predictive modeling of the target of intervention; drug design; and virtual screening; and culminates in the development and implementation of screening methods for potential therapeutics.

"The RNA Institute is an incredible establishment," says University at Albany senior Kathryn Fanning, whose research at the Institute led to a paper in the prestigious Journal of Neuroscience examining the effects of antibiotics known as aminoglycosides on hearing loss. "My work primarily focused on finding evidence that the antibiotics were binding to a specific section of ribosomal RNA to cause the damage."

"My work primarily focused on finding evidence that the antibiotics were binding to a specific section of ribosomal RNA to cause damage."

Since current technologies, instruments, and methods were conceived and designed for DNA and proteins, the Institute—with $15.4 million in funding from the University at Albany and $2.5 million from New York State—is developing, adapting, and re-purposing technologies specifically for their use in RNA-based science. In addition to university and state support, the Institute received $5.37 million from the National Institutes of Health under the 2009 American Recovery and Reinvestment Act (better known as the stimulus bill) to build the physical structure housing the Institute, which was completed in June 2013; $8.13 million in NIH research grants; and $1.3 million from industry partners Sigma Aldrich, Krackeler Scientific, Dell, Bruker Daltonics, and Thermo Fisher Scientific.

The exciting promise of RNA-based therapeutics has enabled the Institute to attract a stellar group of scientists to its advisory board, including Nobel Laureate Ada Yonath of the Weizmann Institute in Israel, and seven members of the National Academy of Sciences, including Dieter Söll and Ron Breaker of Yale University and Gregory J. Hannon of Cold Spring Harbor Laboratory. Researchers at The RNA Institute collaborate with some 350 researchers in more than 50 laboratories worldwide. As New York's national research resource for RNA-based biomedical research, the Institute is committed to initiating and fostering collaborative public-private partnerships, including those involving biotechnology and pharmaceutical companies. It will be a resource for small, medium, and large businesses and aims to spur the development of start-up companies, new R&D centers, and other business operations in New York State.

In collaboration with UAlbany School of Business faculty, The RNA Institute is providing researchers with the tools and strategic know-how to assess the marketplace and successfully develop and commercialize technology in the Capital Region. The RNA Institute will become an economic engine for the state, putting New York at the forefront of cutting-edge RNA technology and fostering unique therapeutic, diagnostic, and technology start-up companies.

Photo: An RNS Institute researcher at work in the lab.